Acute Radiation Syndrome (ARS), also known as radiation toxicity or radiation sickness, is an acute illness that presents after exposure to high levels of radiation, caused by a nuclear accident or attack. It can lead to severe health consequences, including death.
Radiation-induced myelosuppression (RIM) and radiation-induced neutropenia and thrombocytopenia, in particular, is a side effect of anticancer radiotherapy that can cause treatment interruptions and increase the risk of infections and bleeding. Unscheduled interruptions in radiotherapy have been associated with a reduced probability of local tumor control.
Preclinical and clinical studies have shown that orally applied Myelo001 is effective in reducing hematopoietic symptoms caused by radiation overexposure or radio-chemotherapy. The effect of Myelo001 was observed for several distinct hematopoietic cell lineages including neutrophils, lymphocytes, and thrombocytes. Comprehensive chronic toxicology and safety studies, as well as clinical studies, have confirmed an excellent safety profile of Myelo001.
Myelo001 is currently investigated in non-clinical studies as a prophylactic and therapeutic treatment for acute radiation syndrome. Data published in blood showed that Myelo001 treatment leads to a significant reduction in mortality in an ARS animal model.
The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Myelo001 for the treatment of Acute Radiation Syndrome (ARS).